Efficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome

Fragile X Syndrome Clinical Trial

— Harbor-C  

Official title:

A Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Children With Fragile X Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01325220
• Other study ID #: 209FX302
• Status: Completed
• Phase: Phase 3
• First received: March 28, 2011
• Last updated: July 30, 2013
• Start date: June 2011

Study information

• Verified date: July 2013
• Source: Seaside Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

There will be four study periods: Screening (up to 14 days in length), the Treatment Period (8 weeks), the Withdrawal Period (22 days), and Follow-up Period (up to 31 days). Subjects will be randomized to receive either STX209 (5 mg twice daily [BID], 10 mg BID or 10 mg three times daily [TID]) or placebo. Efficacy,safety and tolerability assessments will be performed periodically

Recruitment information / eligibility

• Status: Completed
• Enrollment: 172
• Est. primary completion date: June 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 5 Years to 11 Years

Eligibility

Inclusion Criteria:

• Molecular documentation of the full FMR1 mutation

• Current pharmacological treatment regimen has been stable for at least 4 weeks prior to Screening.

• Subjects with a history of seizure disorder must currently be receiving treatment with antiepileptics and must have been seizure free for 6 months, or must be seizure free for 3 years if not currently receiving antiepileptics.

• If the subject is already receiving stable non-pharmacologic educational, behavioral, and/or dietary interventions, participation in these programs must have been continuous during the 2 months prior to Screening

Exclusion Criteria:

• Subjects with any condition, including alcohol and drug abuse, which might interfere with the conduct of the study, confound interpretation of the study results, or endanger their own well-being.

• Subjects who plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study.

• Subjects who have taken another investigational drug within the last 30 days.

• Subjects who are not able to take oral medications

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• arbaclofen
5 mg bid
• arbaclofen
10 mg bid
• arbaclofen
10 mg tid
• Placebo
tid

Locations

Country	Name	City	State
United States	Akron Children's Hospital	Akron	Ohio
United States	University of Colorado Denver, Children's Hospital	Aurora	Colorado
United States	Kennedy Krieger Institute	Baltimore	Maryland
United States	Rush University Medical Center	Chicago	Illinois
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	University of Missouri, Thompson Research Center for Autism & Neurodevelopmental Disorders	Columbia	Missouri
United States	Childrens Medical Center Dallas	Dallas	Texas
United States	Emory University School of Medicine	Decatur	Georgia
United States	Children's Hospital of Michigan	Detriot	Michigan
United States	Duke Clinical Research Unit	Durham	North Carolina
United States	Kansas University Clinical Research Center	Fairway	Kansas
United States	Greenwood Genetics Center	Greenwood	South Carolina
United States	Red Oaks Psychiatry Associates, P.A.	Houston	Texas
United States	Texas Children's Hospital	Houston	Texas
United States	Miller Children's Hospital	Long Beach	California
United States	Suburban Research Associates/Elwyn Genetics	Media	Pennsylvania
United States	University of Miami, Mailman Center for Child Development	Miami	Florida
United States	Vanderbilt Kennedy Center	Nashville	Tennessee
United States	Seaver Autism Center, Mount Sinai Medical Center	New York	New York
United States	University of Oklahoma, Physician's Child Study Center	Oklahoma City	Oklahoma
United States	Lake Mary Pediatrics	Orange City	Florida
United States	Southwest Autism Research & Resource Center	Phoenix	Arizona
United States	University of California-Davis, M.I.N.D. Institute	Sacramento	California
United States	Road Runner Research	San Antonio	Texas
United States	Psychiatric Centers at San Diego	San Diego	California
United States	Seattle Children's Hospital	Seattle	Washington
United States	New York State Institute for Basic Research in Developmental Disabilities	Staten Island	New York
United States	University of Massachusetts	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Seaside Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Aberrant Behavior Checklist-Lethargy Social Withdrawal subscale	This is a single subscale of the aberrant behavior checklist entitiled Lethargy Social Withdrawal	at 8 weeks of treatment	No